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Introduction

Fanconi anemia (FA) is a rare genetic disease. Failure of blood stem cells to produce sufficient numbers of mature blood cells, as well as progression to myelodysplastic syndrome (MDS) and acute myelogenous leukemia (AML) are the greatest risk factors of morbidity and mortality in these patients. Treatment options are limited in FA. For the majority of patients, stem cell transplantation is the ultimate therapy, but donor availability, graft failure, and FA-specific transplant toxicities are still significant hurdles towards a curative treatment of FA-associated blood manifestations.

This clinical trial proposes to treat patients with FA for 6 months with a research medication (eltrombopag) to assess safety and efficacy at improving blood counts in FA. Eltrombopag is an oral drug designed to mimic a protein called thrombopoietin (TPO). This protein is necessary for normal blood production and may not function in patients with FA. Responders at 6 months will be able to continue eltrombopag on the extension part of this protocol for an additional 3 years. During this time frame, we anticipate further improvement of peripheral blood cell counts that will eventually result in the discontinuation of eltrombopag after a tapering period.

To get started:

  • Navigate through the menu to learn more about the study.
  • Log in to the application and fill out questionnaires.

Please contact us if you have any questions or concerns.